Advancing New Chemical Modalities into Clinical Studies

Drug discovery and development has experienced an incredible
paradigm
shift in the past two decades. What once was considered a predominant
R&D landscape of small molecules within a prescribed properties
and mechanism space now includes an innovative wave of new chemical
modalities. Scientists in the pharmaceutical industry can now strategize
across a variety of modalities to find the best option to modulate
a given target and provide treatment for a specific disease. We have
witnessed a remarkable change not only in molecular design but also
in creative approaches to drug delivery that have enabled advancement
of novel modalities to clinical studies. In this Microperspective,
we evaluate the critical differences between traditional small molecules
and beyond rule of 5 compounds, peptides, oligonucleotides, and biologics
for advancing into development, particularly their pharmacokinetic
profiles and drug delivery strategies.